Welcome to Gene Therapy Net
Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.
Gene therapy delivered to the brain shows promise in children with rare neurodegenerative disease
Posted on: 16 July 2021, source: FierceBiotech
Scientists from Ohio State University have developed a novel method for delivering gene therapy to specific regions of the brain. Now, they have evidence from a small clinical trial in children that the treatment could address a rare, inherited neurodegenerative disease. And they believe their technique could eventually be used to treat more common brain diseases, like Alzheimer’s and Parkinson’s. The Ohio State team developed the gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency, which hampers the body’s ability to make dopamine and serotonin and results in developmental delays and a range of motor and behavioral symptoms. The gene therapy uses a viral vector to carry DNA-expressing AADC to the brain.
Scientists from Ohio State University have developed a novel method for delivering gene therapy to specific regions of the brain. Now, they have evidence from a small clinical trial in children that the treatment could address a rare, inherited neurodegenerative disease. And they believe their technique could eventually be used to treat more common brain diseases, like Alzheimer’s and Parkinson’s. The Ohio State team developed the gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency, which hampers the body’s ability to make dopamine and serotonin and results in developmental delays and a range of motor and behavioral symptoms. The gene therapy uses a viral vector to carry DNA-expressing AADC to the brain.
A year after getting UniQure's gene therapy, hemophilia patients are still doing better
Posted on: 23 June 2021, source: BioPharma Dive
One year after receiving an experimental gene therapy developed by the Dutch drugmaker UniQure, patients with hemophilia B aren't having nearly as many bleeding issues as they used to have. In hemophilia, genetic mutations prevent the body from making proteins needed to clot blood. People with the less common, "B" form of the disorder are missing a protein known as Factor IX. UniQure's medicine is meant to provide a working version of the gene so patients can generate their own clotting protein, and rely less — or ideally, not at all — on so-called replacement factor treatments.
One year after receiving an experimental gene therapy developed by the Dutch drugmaker UniQure, patients with hemophilia B aren't having nearly as many bleeding issues as they used to have. In hemophilia, genetic mutations prevent the body from making proteins needed to clot blood. People with the less common, "B" form of the disorder are missing a protein known as Factor IX. UniQure's medicine is meant to provide a working version of the gene so patients can generate their own clotting protein, and rely less — or ideally, not at all — on so-called replacement factor treatments.
Decades after a tragic failure, gene therapy successfully treats a rare liver disease
Posted on: 19 May 2021, source: Science
Twenty-two years ago, one of scientists’ first attempts at gene therapy ended in tragedy when a young man died. The story of Jesse Gelsinger, who had a rare liver disorder, became a textbook example of irresponsible medical research. For years, the case hobbled efforts to treat diseases by adding new DNA to a sick person’s cells. Now, a fresh effort to cure Gelsinger’s disease is bearing fruit, in the latest sign of the field’s resurgence.
Twenty-two years ago, one of scientists’ first attempts at gene therapy ended in tragedy when a young man died. The story of Jesse Gelsinger, who had a rare liver disorder, became a textbook example of irresponsible medical research. For years, the case hobbled efforts to treat diseases by adding new DNA to a sick person’s cells. Now, a fresh effort to cure Gelsinger’s disease is bearing fruit, in the latest sign of the field’s resurgence.
New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy
Posted on: 4 May 2021, source: News-Medical.net
UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.
UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.
New approach to CAR T gene therapy yields efficient and longer-lasting response against HIV
Posted on: 5 April 2021, source: News-Medical.net
A UCLA research team has shown that using a truncated form of the CD4 molecule as part of a gene therapy to combat HIV yielded superior and longer-lasting results in mouse models than previous similar therapies using the CD4 molecule. This new approach to CAR T gene therapy -- a type of immunotherapy that involves genetically engineering the body's own blood-forming stem cells to create HIV-fighting T cells -- has the potential to not only destroy HIV-infected cells but to create "memory cells" that could provide lifelong protection from infection with the virus that causes AIDS.
A UCLA research team has shown that using a truncated form of the CD4 molecule as part of a gene therapy to combat HIV yielded superior and longer-lasting results in mouse models than previous similar therapies using the CD4 molecule. This new approach to CAR T gene therapy -- a type of immunotherapy that involves genetically engineering the body's own blood-forming stem cells to create HIV-fighting T cells -- has the potential to not only destroy HIV-infected cells but to create "memory cells" that could provide lifelong protection from infection with the virus that causes AIDS.
EMA Launches Safety Review After Gene Therapy Trial Patient Develops Blood Cancer
Posted on: 13 March 2021, source: BioSpace
The European Medicines Agency (EMA) has launched a safety review of bluebird bio’s thalassaemia drug Zynteglo, a conditionally licensed gene therapy in Europe. This review comes after researchers identified an acute myeloid leukaemia (AML) case in a patient who received bb1111 (LentiGlobin), an investigational gene therapy for sickle cell disease (SCD), in a Phase I/II study. This therapy relies on the same viral vector found in Zynteglo which delivers a gene into cells.
The European Medicines Agency (EMA) has launched a safety review of bluebird bio’s thalassaemia drug Zynteglo, a conditionally licensed gene therapy in Europe. This review comes after researchers identified an acute myeloid leukaemia (AML) case in a patient who received bb1111 (LentiGlobin), an investigational gene therapy for sickle cell disease (SCD), in a Phase I/II study. This therapy relies on the same viral vector found in Zynteglo which delivers a gene into cells.
Gene therapy trials for sickle cell disease halted after two patients develop cancer
Posted on: 22 February 2021, source: Science
A company has stopped its clinical studies of a promising gene therapy for the blood disorder sickle cell disease after two people who participated developed leukemia-like cancer. Bluebird bio is now investigating whether a virus it uses to deliver a therapeutic gene caused the cancers, reviving old concerns about the risks of this approach. It’s also possible the cancers stemmed from chemotherapy the patients received to prepare their bodies for the gene’s delivery. “This is really a sad development whatever the cause,” says Donald Kohn of the University of California, Los Angeles, who has led gene therapy trials for sickle cell and other diseases.
A company has stopped its clinical studies of a promising gene therapy for the blood disorder sickle cell disease after two people who participated developed leukemia-like cancer. Bluebird bio is now investigating whether a virus it uses to deliver a therapeutic gene caused the cancers, reviving old concerns about the risks of this approach. It’s also possible the cancers stemmed from chemotherapy the patients received to prepare their bodies for the gene’s delivery. “This is really a sad development whatever the cause,” says Donald Kohn of the University of California, Los Angeles, who has led gene therapy trials for sickle cell and other diseases.
Gene Therapy News
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Gene Therapy - AroundtheO
Aug 3, 2021 | 22:48 pm
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FDA allows Novartis gene therapy trials to resume after nearly two year pause - BioPharma Dive
Aug 3, 2021 | 18:32 pm
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New 'Universal Switch' Lets Scientists Fine-Tune Gene Therapy - Singularity Hub
Aug 3, 2021 | 09:00 am
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Targeting gene therapy for achromatopsia - Ophthalmology Times
Aug 3, 2021 | 06:38 am
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CDMOs Gear Up to Churn Out Cell and Gene Therapies - Genetic Engineering & Biotechnology News
Aug 2, 2021 | 08:55 am
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